Gene Based Pharmaceuticals and Gene Therapy

New classes of products are being tested for use in humans and animals, all sharing genes as common targets. Products based on antisense technology directed toward neutralizing messenger RNA are probably being pursued most vigorously; gene therapy through permanent alteration of chromosomes might hold the greatest potential for treatment of diseases like cancer and for correction of genetic disease. The products depend either on classes of compounds that are related to nucleic acids (oligonucleotides and oligonucleotide analogues), on cells that have been genetically altered, of on viruses that bear appropriate nucleic acids.

For the large-scale production of nucleotides and nucleotide analogues, new molecular techniques must be developed. There are now no procedures for making substantial quantities of these types of materials in high purity and with appropriate chirality. Basic chemical and biochemical techniques must be developed for their preparation; new techniques (probably based on high-pressure chromatography) will be required for large-scale purification, and biological methods might be required for preparation of precursors and perhaps for formation of bonds.

For genetically modified cells and viruses, the usual techniques for mammalian-cell culture and molecular biology will be required, as will additional measures for safety and for economical, patient-specific production.

Tags: Bio Technology, Bio Genetics, Bio Process Engineering

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